The Use of High Resolution Chest Computed Tomography in Alpha-1 Antitrypsin Deficiency (QUANTUM-1)

This study has been completed.

Sponsor:

Medical University of South Carolina

ClinicalTrials.gov Identifier:

NCT00532805

First Posted: September 20, 2007

Last Update Posted: December 19, 2012

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government.
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Individuals with a deficiency of alpha-1 antitrypsin (AAT) often develop emphysema. Traditional lung function tests may not be the most accurate way to measure the progression of emphysema. This study will compare high resolution computed tomography (CT) scans to spirometry to measure the progression of emphysema.

Further study details as provided by Charlie Strange, Medical University of South Carolina:

Primary Outcome Measures:

CT density slope [ Time Frame: 3 years ]

Biospecimen Retention: Samples With DNA

50 cc of serum at visits baseline, 6 months, 12 months, 18 months, 24 months and 36 months will be retained. These are kept at the University of Florida in the laboratory of Dr. Mark Brantly.

There is an associated but independent DNA collection that is done if the patient is willing through an independent study and consent process with the University of Florida Alpha-1 DNA and Tissue Bank. This is a public resource with a scientific advisory committee with samples available for researcher access.

AAT deficiency is a genetic disorder associated with emphysema. Spirometry, the lung function test that measures how well the lungs exhale air, is used to diagnose and track the progression of emphysema. Some studies have suggested that forced expiratory volume in 1 second (FEV1) measurements, a type of spirometry test, may lack accuracy in detecting disease progression in cases of severe AAT deficiency. Another method, high resolution chest CT scans, may be more accurate at measuring the progression of emphysema. The purpose of this study is to determine if high resolution CT scans are better at detecting the progression of emphysema than lung function tests. Results from this study may lead to the development of a more accurate way to assess lung tissue loss and may improve the understanding of lung destruction in AAT deficiency.

This study will last 4 years and will enroll people with AAT deficiency who have nearly normal lung function test results. Study visits, each lasting about 4 hours, will occur at baseline and months 6, 12, 18, 24, and 36. At each visit, participants will undergo lung function tests, a CT scan, blood collection, and a physical exam. Female participants will have urine collected for a pregnancy test. All participants will also complete questionnaires to assess health status and lung function. Study researchers will call participants every 2 months to collect information on lung disease symptoms and medication changes.

Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:

18 Years and older (Adult, Senior)

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

No

Sampling Method:

Non-Probability Sample

Study Population

Individuals with alpha-1 antitrypsin who have nearly normal lung function tests. Participants will be identified from patients of the investigators, physician referral, and the Alpha-1 Foundation Research Registry.

Criteria

Inclusion Criteria:

Diagnosis of AAT deficiency, as determined by both of the following conditions:

Required to take any of the following medications within 48 hours of scheduled lung function testing: dicyclomine (Bentyl), propantheline (Pro-Banthine), mepenzolate (Cantil), methscopolamine (Pamine), and scopolamine (Transderm-Scop)

Known allergy or intolerance to tiotropium or albuterol

Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00532805

Locations

United States, Colorado

National Jewish Medical and Research Center

Denver, Colorado, United States

United States, Florida

University of Florida Medical Center

Gainesville, Florida, United States

United States, Massachusetts

Harvard/Brigham and Women's Hospital

Boston, Massachusetts, United States

United States, Ohio

Cincinnati Children's Medical Center

Cincinnati, Ohio, United States

Cleveland Clinic Foundation

Cleveland, Ohio, United States

United States, Oregon

Oregon Health and Sciences University

Portland, Oregon, United States

United States, South Carolina

Medical University of South Carolina

Charleston, South Carolina, United States

Sponsors and Collaborators

Medical University of South Carolina

National Center for Research Resources (NCRR)

Alpha-1 Foundation

Investigators

Study Chair:

Charlie Strange, MD

Medical University of South Carolina

More Information

Responsible Party:

Charlie Strange, Professor of Pulmonary Medicine, Medical University of South Carolina